Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Fabry Disease Clinical Trial

Official title:

A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00837824
• Other study ID #: AGAL-022-02
• Status: Terminated
• Phase: Phase 2
• First received: October 23, 2008
• Last updated: March 19, 2015
• Start date: December 2002
• Est. completion date: August 2003

Study information

• Verified date: March 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 20
• Est. completion date: August 2003
• Est. primary completion date: August 2003
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

Inclusion Criteria:

• provided written informed consent prior to any study-related procedures being performed.

• be =16 years old.

• have a current diagnosis of Fabry disease (defined as abnormal a-galactosidase (a GAL) enzyme levels or Fabry genotype).

• have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.

• have the ability to comply with the requirements of the protocol

• have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

• if they did not meet the specific inclusion criteria.

• if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.

• had previously received enzyme replacement therapy (ERT) for their Fabry disease.

• had diabetic nephropathy.

• were pregnant or lactating.

• were unwilling to comply with the requirements of the protocol.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Chronic Kidney Disease, Stage IV (Severe)
• Fabry Disease
• Kidney Diseases
• Renal Insufficiency, Chronic

Intervention

Biological:
• Fabrazyme (agalsidase beta)
1.0 mg/kg every 2 weeks
• Fabrazyme (agalsidase beta)
3.0 mg/kg every 2 weeks

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company	CRL/Medinet

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease	The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.	7 months	No
Secondary	Plasma Globotriaosylceramide (GL-3)	This outcome measure evaluated the mean plasma GL-3 values for all patients to see if it decreased while on Fabrazyme. Normal plasma GL-3 level is defined as = 7.03 µg/mL.	Evaluated at Baseline, Month 3, and Final Visit	No